Targeted Treatment of Soft-Tissue Sarcoma
<b>Background</b>: Soft-tissue sarcoma (STS) is a heterogeneous group of sarcomas with a low incidence. The treatment of advanced disease is poor, and mortality is high. We aimed to generate an overview of the clinical experiences with targeted treatments based on a pre-specified target...
| Main Authors: | , , , |
|---|---|
| Format: | Article |
| Language: | English |
| Published: |
MDPI AG
2023-04-01
|
| Series: | Journal of Personalized Medicine |
| Subjects: | |
| Online Access: | https://www.mdpi.com/2075-4426/13/5/730 |
| _version_ | 1797599549343137792 |
|---|---|
| author | Anne Iren Riskjell Vivi-Nelli Mäkinen Birgitte Sandfeld-Paulsen Ninna Aggerholm-Pedersen |
| author_facet | Anne Iren Riskjell Vivi-Nelli Mäkinen Birgitte Sandfeld-Paulsen Ninna Aggerholm-Pedersen |
| author_sort | Anne Iren Riskjell |
| collection | DOAJ |
| description | <b>Background</b>: Soft-tissue sarcoma (STS) is a heterogeneous group of sarcomas with a low incidence. The treatment of advanced disease is poor, and mortality is high. We aimed to generate an overview of the clinical experiences with targeted treatments based on a pre-specified target in patients with STS. <b>Methods</b>: A systematic literature search was conducted in PubMed and Embase databases. The programs ENDNOTE and COVIDENCE were used for data management. The literature was screened to assess the article’s eligibility for inclusion. <b>Results</b>: Twenty-eight targeted agents were used to treat 80 patients with advanced STS and a known pre-specified genetic alteration. MDM2 inhibitors were the most-studied drug (<i>n</i> = 19), followed by crizotinib (<i>n</i> = 9), ceritinib (<i>n</i> = 8), and <sup>90</sup>Y-OTSA (<i>n</i> = 8). All patients treated with the MDM2 inhibitor achieved a treatment response of stable disease (SD) or better with a treatment duration of 4 to 83 months. For the remaining drugs, a more mixed response was observed. The evidence is low because most studies were case reports or cohort studies, where only a few STS patients were included. <b>Conclusions</b>: Many targeted agents can precisely target specific genetic alterations in advanced STS. The MDM2 inhibitor has shown promising results. |
| first_indexed | 2024-03-11T03:35:54Z |
| format | Article |
| id | doaj.art-cc8852e67a0246769f889b00e8647975 |
| institution | Directory Open Access Journal |
| issn | 2075-4426 |
| language | English |
| last_indexed | 2024-03-11T03:35:54Z |
| publishDate | 2023-04-01 |
| publisher | MDPI AG |
| record_format | Article |
| series | Journal of Personalized Medicine |
| spelling | doaj.art-cc8852e67a0246769f889b00e86479752023-11-18T02:03:18ZengMDPI AGJournal of Personalized Medicine2075-44262023-04-0113573010.3390/jpm13050730Targeted Treatment of Soft-Tissue SarcomaAnne Iren Riskjell0Vivi-Nelli Mäkinen1Birgitte Sandfeld-Paulsen2Ninna Aggerholm-Pedersen3Faculty of Health, Aarhus University, 8000 Aarhus C, DenmarkFaculty of Health, Aarhus University, 8000 Aarhus C, DenmarkDepartment of Clinical Biochemistry, Viborg Regional Hospital, 8800 Viborg, DenmarkDepartment of Oncology, Aarhus University Hospital, Palle Juul-Jensens Boulevard 99, 8200 Aarhus N, Denmark<b>Background</b>: Soft-tissue sarcoma (STS) is a heterogeneous group of sarcomas with a low incidence. The treatment of advanced disease is poor, and mortality is high. We aimed to generate an overview of the clinical experiences with targeted treatments based on a pre-specified target in patients with STS. <b>Methods</b>: A systematic literature search was conducted in PubMed and Embase databases. The programs ENDNOTE and COVIDENCE were used for data management. The literature was screened to assess the article’s eligibility for inclusion. <b>Results</b>: Twenty-eight targeted agents were used to treat 80 patients with advanced STS and a known pre-specified genetic alteration. MDM2 inhibitors were the most-studied drug (<i>n</i> = 19), followed by crizotinib (<i>n</i> = 9), ceritinib (<i>n</i> = 8), and <sup>90</sup>Y-OTSA (<i>n</i> = 8). All patients treated with the MDM2 inhibitor achieved a treatment response of stable disease (SD) or better with a treatment duration of 4 to 83 months. For the remaining drugs, a more mixed response was observed. The evidence is low because most studies were case reports or cohort studies, where only a few STS patients were included. <b>Conclusions</b>: Many targeted agents can precisely target specific genetic alterations in advanced STS. The MDM2 inhibitor has shown promising results.https://www.mdpi.com/2075-4426/13/5/730targeted treatmentgene mutationssarcoma |
| spellingShingle | Anne Iren Riskjell Vivi-Nelli Mäkinen Birgitte Sandfeld-Paulsen Ninna Aggerholm-Pedersen Targeted Treatment of Soft-Tissue Sarcoma Journal of Personalized Medicine targeted treatment gene mutations sarcoma |
| title | Targeted Treatment of Soft-Tissue Sarcoma |
| title_full | Targeted Treatment of Soft-Tissue Sarcoma |
| title_fullStr | Targeted Treatment of Soft-Tissue Sarcoma |
| title_full_unstemmed | Targeted Treatment of Soft-Tissue Sarcoma |
| title_short | Targeted Treatment of Soft-Tissue Sarcoma |
| title_sort | targeted treatment of soft tissue sarcoma |
| topic | targeted treatment gene mutations sarcoma |
| url | https://www.mdpi.com/2075-4426/13/5/730 |
| work_keys_str_mv | AT anneirenriskjell targetedtreatmentofsofttissuesarcoma AT vivinellimakinen targetedtreatmentofsofttissuesarcoma AT birgittesandfeldpaulsen targetedtreatmentofsofttissuesarcoma AT ninnaaggerholmpedersen targetedtreatmentofsofttissuesarcoma |